Advances in gene therapy technologies to treat retinitis pigmentosa.
Advances in gene therapy technologies to treat retinitis pigmentosa.
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DOI：
10.2147/opth.s38041
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发表时间：
2014
期刊：
影响因子：
--
通讯作者：
Linden R
中科院分区：
文献类型：
--
作者：
Petrs-Silva H;Linden R
视网膜色素变性是导致视网膜进行性变性的一类疾病。近年来，遗传性视网膜营养不良的基因治疗的实验方法取得了进展，包括将基因安全地输送到人类视网膜。本文综述了利用重组腺相关病毒载体进行视网膜病变基因治疗的研究进展，该载体具有良好的安全性，目前已在多个先天性视网膜疾病的临床试验中获得成功。RP的基因治疗正在各种动物模型中进行，其结果提高了对未来临床应用的期望。尽管如此，将这些策略转换到床边需要进一步了解导致视觉缺陷的突变和机制，以及彻底检查潜在的不良影响。
Retinitis pigmentosa (RP) is a class of diseases that leads to progressive degeneration of the retina. Experimental approaches to gene therapy for the treatment of inherited retinal dystrophies have advanced in recent years, inclusive of the safe delivery of genes to the human retina. This review is focused on the development of gene therapy for RP using recombinant adenoassociated viral vectors, which show a positive safety record and have so far been successful in several clinical trials for congenital retinal disease. Gene therapy for RP is under development in a variety of animal models, and the results raise expectations of future clinical application. Nonetheless, the translation of such strategies to the bedside requires further understanding of the mutations and mechanisms that cause visual defects, as well as thorough examination of potential adverse effects.